(RTTNews) - Prime Medicines Inc. (PRME), a biotechnology company, on Monday announced the receipt of a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for PM359, a hematopoietic stem cell therapy for p47phox deficiency chronic granulomatous disease.
Chronic granulomatous disease (CGD) is a rare inherited hematologic disorder characterized by susceptibility to severe infections alongside inflammatory and autoimmune complications. The disease affects the production of functional neutrophils and is diagnosed with a frequency of about one in every 100,000-200,000 individuals in the U.S. alone with the p47phox accounting for 25% of all cases.
PM359 is an investigational autologous hematopoietic stem cell therapy designed to correct the delGT mutation in NCF1, the most prevalent disease-causing mutation in p47phox-deficient CGD. In addition to RMAT designation, PM359 has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
The company is currently evaluating PM359 in a multinational, first-in-human Phase 1/2 trial, with initial data providing support for the RMAT designation. The company plans to align with the FDA to advance studies facilitating a future biologics license application (BLA) submission.
If approved, the treatment may be the first approved gene therapy for treating CGD.
PRME is currently trading at $3.43, up 6.35%.
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